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Wednesday, May 28, 2014
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Back To The Future: The Revenge Of Gene Therapy

May 28, 2014

By Karl Thiel

I remember being hugely excited about gene therapy back in the 1990s. The idea of using an engineered virus to shuttle corrected genes directly into the genome was elegant and appealing, and came with the (perhaps naive) hope that once the vectors were worked out, there was plenty of low-hanging fruit that could be addressed through an essentially plug-and-play approach with different genes. More...


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Pirfenidone has gone down a long road that could finally lead to approval for idiopathic pulmonary fibrosis (IPF). First discovered in 1976, InterMune has been developing the drug for IPF since 2007, but got an FDA rejection back in 2010. New promising phase 3 data just published in the New England Journal of Medicine and presented at the American Thoracic Society could turn the tide. Still, InterMune isn't alone. Pivotal data on Boehringer Ingelheim's nintedanib, a competing IPF drug, were published in the same issue. While pirfenidone arguably looks a little better, the difference isn't huge, so the company's may end up sharing the market. (And then there's FibroGen's IPF drug, which recently produced phase 2 results.)

Isis, meanwhile, is trying to keep the momentum for antisense drugs going, announcing positive clinical news for its venous thrombosis and diabetes drugs, two more in a series of clinical announcements coming out of the company's extensive pipeline. -KT
Isis (ISIS) Surges As Blood Thinner Drug ISIS-FXI Succeeds In Ph2 Trial; Stock Up 7.54% At Market Close (May 22, 2014)

Gilead (GILD)'s RSV Drug GS-5806 Wows In Ph2 Trial

Lilly (LLY)'s Necitumumab Improves Overall Survival In Largest Ever Ph3 Study In First-Line Tx Of Stage IV Squamous Non-Small Cell Lung CA

Vertex (VRTX) Release: Addition Of VX-661 To KALYDECO® (Ivacaftor) Improves Lung Function In People With CF Who Are Heterozygous For The F508del And G551D Mutations In 28-Day Ph2 Proof-Of-Concept Study

InterMune (ITMN) Rises After Fatal Lung Disease Drug Slows Damage In Ph3 Study

More Bench To Clinic News
There have been some significant changes among the top brass at Celgene, including a new CFO and promotions from within to the positions of President and COO (Mark Alles), and the heads of Inflammation & Immunology (Scott Smith) and Hematology & Oncology (Jacqualyn Fouse). There are doubtless some thoughts of succession in these changes, with Mark Alles and Jacqualyn Fouse looking like the top candidates. But at age 59, CEO Robert Hugin is likely to hang around for a few years yet.

Activists trying to link Gilead chief John Martin's compensation to drug access didn't get very far, winning only 2% of shareholder votes. But Michael Weinstein of the AIDS Healthcare Foundation, who was responsible for the proposal, plans to continue his campaign to get Gilead to lower its drug prices. -KT
Organization Changes At Celgene (CELG) Have Got Investors Wondering Who Will Take Over Once Hugin Steps Down

AcelRx Pharma (ACRX) Triples Headcount, Expands Bay Area Facility

Gilead (GILD) Shareholders Decimate Plan To Tie CEO Compensation To Drug Access

Teva (TEVA) To Speed Up Cost-Cutting Plans, Layoffs And Facility Shutdowns On The Horizon

Alcon (ACL) Slashes Headcount By 200

More Career Track News
Well, it looks like a Pfizer-AstraZeneca merger won't be happening, at least not in this round. Pfizer has failed to increase its offer after being rejected by Astra's board, saying its final offer "fully valued" the company. The strategy here is likely to see if Astra's shareholders, disappointing by the end of talks, will pressure the company to play ball with Pfizer. After a three-month cooling off period, talks could recommence.

Valeant is also having trouble finding love. It has said it will up its bid for Allergan from the current $46 billion sometime this week, but how much sugar will Valeant have to add to its $153 per share offer? According to a J.P. Morgan survey, $180 to $200 a share—a pretty steep rise from here. -KT
Astra (AZN) Rejects Pfizer (PFE)'s Final Offer

Ophthotech (OPHT) May Pocket $1B From Eye Drug Deal With Novartis (NVS), Ophthotech Stock Up +24.48% At Market Close (May 20, 2014)

Lundbeck (LUN.CO) Swallows Up Chelsea Therapeutics (CHTP) In Deal Worth $658M

Neurotez, GCA Therapeutics Strike $102.5M+ Alzheimer's Pact

Valeant (VRX) Will Sweeten $46B Allergan (AGN) Bid

More Golden Handshakes News

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• Genetown (Lebanon, New Hampshire)

Vice President of Protein Sciences
Seeking an accomplished scientific leader to join its management team as Vice President of Protein Sciences. The Vice President of Protein Sciences will report to the Chief Scientific Officer and lead the High Throughput Expression and Protein Analytics groups.

• Biotech Bay (Newark, California)

Chief Commercial Officer
He/she will be responsible for leading the development and execution of the company's commercial strategy across the company's portfolio of biopharmaceutical products. The Chief Commercial Officer will have responsibility for assessing, continuing to build, managing and developing a high-performance Commercial organization.

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HUYA Bioscience
Vice President of Oncology
Will Identify new product in-licensing, out-licensing, and partnering opportunities which align with the strategic objectives of HUYA Bioscience International. The VP of Business Development will execute on current and long-range business strategies to grow the value of the organization.

• BioCapital (Blacksburg, Virginia)

Vice President, Ultra Vector Division
Is responsible for leading the strategic and tactical planning and the operations management of the organization's DNA design, production and testing activities. The role is responsible for the DNA production and the Ultra Vector platform which is the core competitive advantage of the company.

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If you follow biotech stocks, you know that a sizzling hot market turned sharply southward in March. I think I prematurely wondered if the biotech IPO window might be closing a few months back, so I'm loathe to make another prediction. I'll limit myself to observing that several more companies have postponed IPOs or slashed prices to get out. Then again, Radius Health canceled its IPO plans for the second time in two years, only to come back swinging a couple weeks later. This may have legs yet. -KT
Fremont-Based Ardelyx Files For $69M IPO

San Francisco Biotech Startup Nurix Taps $25.1M Financing

Santa Monica's Kite Pharma Gears Up For IPO Debut

Gene Therapy Biotech Lysogene Reels In $22M

Kymab Locks Down $40M For Antibody Research

More Money Talk News

Teva Pharmaceutical didn't have much chance of winning its suit against FDA, so it's no surprise that its latest effort to block launch of a generic competitor to its multiple sclerosis drug Copaxone came up short. But the judge did leave the door open for Teva to come back and seek an injunction once a generic product is actually approved. So Mylan and Momenta Pharmaceuticals can't rest easy quite yet. -KT
Mylan (MYL) Applauds Court's Ruling Dismissing Teva (TEVA)'s Suit Against FDA On Copaxone®

Glaxo (GSK) Dodged Millions In China Drug Tax Scam

SEC Accuses Two Doctors Of Insider Trading After GTx (GTXI) Clinical Trial Is Halted

Jury Finds For Takeda (TKPYY) In Diabetes Drug Case

Lilly (LLY) Loses Major Patent Case To Actavis (ACT)

More Legal Briefs News
Two new molecular entities were approved in May (bringing us up to 14 year-to-date). Takeda's Entyvio was approved for ulcerative colitis and Crohn's disease in patients who haven't gotten adequate relief from other treatments, including TNF drugs Humira and Remicade. As such, it may have a bright future, particularly as it should be safer than the TNF drugs. But it has to be given by intravenous infusion, one reason analysts are looking at peak sales of around $500 million. Merck, and Schering-Plough before it, had hoped Zontivity (Vorapaxar) might become a megablockbuster with peak sales of $5 billion or better. But side effects found in the clinical trials—namely a risk of intracranial bleeding in patients with a history of stroke—seem likely to put a serious lid on sales. Merck has already written off $1.7 billion in R&D costs for the drug, so expectations aren't nearly what they once were. -KT
Takeda (TKPYY)'s Entyvio Wins FDA Approval For Severe Colitis

Clovis Oncology (CLVS) Wins Breakthrough Status For Lung CA Candidate CO-1686

Merck (MRK) Release: FDA Approves ZONTIVITY™ (Vorapaxar), First-In-Class PAR-1 Antagonist, For The Reduction Of Thrombotic Cardiovascular Events In Pts With A History Of Heart Attack Or With Peripheral Arterial Disease

Astra (AZN) Gets A Lift In Pfizer (PFE) Fight As FDA Approves Heart Pill Epanova

Glaxo (GSK) Touts Positive Incruse™-Ellipta®-Advair®-Diskus® Combo Data

More Rockville Files News

Karl Thiel is an analyst for The Motley Fool and a longtime follower of the biotech industry. He lives in Austin, Texas.

You may contact Karl Thiel at

BioSpace, Inc.
6465 South Greenwood Plaza, Suite 400
Centennial, Colorado 80111 U.S.A.
Phone: 877-277-7585
Fax: 800-595-2929
BioSpace, Inc.
90 New Montgomery Street, Suite 414
San Francisco, California 94105 U.S.A.
Phone: 877-277-7585
Fax: 415-576-9217

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